Our Publications

Publications

Medical Matters

Family Matters -
Summer 2009

Pediatric Research Spotlighted at National Conference

Each year, top specialists from around the world who treat childhood cancer participate in the American Society of Pediatric Hematology/Oncology’s (ASPHO) annual meeting. Once again, experts from the Children’s Cancer Hospital at M. D. Anderson were selected to present their cancer research at the meeting.

Alejandro Levy, M.D., fellow at the Children’s Cancer Hospital, kicked things off with one of the first presentations. His research, under the direction of assistant professor Patrick Zweidler-McKay, M.D., Ph.D., found a new drug that restricts the growth in pre-clinical tests of neuroblastoma, a childhood brain cancer.

The study, conducted with human neuroblastoma cells transplanted into mice, showed how 3-BrOP, a glycolysis inhibitor, starved the cancer cells to death by shutting down their main energy source, glucose.

“As we explore alternative options to standard chemotherapy agents, we are finding drugs, like 3-BrOP, that have the potential to destroy cancer cells while leaving healthy cells unharmed,” said, Zweidler-McKay. “These drugs can often enhance the efficacy of other treatments, potentially leading to more successful combinations and better outcomes for our young patients.”

Zweidler-McKay also presented research on neuroblastoma that he led. From his research, he discovered that the drug, AMD3100, hinders the formation of tumor blood vessels and reduces human neuroblastoma tumor growth by more than 75 percent in mice.

“AMD3100 works by shutting down the process that tumors need to set up vascular systems,” said Zweidler-McKay. “The drug doesn’t kill neuroblastoma cells directly, but it prevents tumors from growing rapidly by disrupting their blood supply.”

First-Ever Trial Opened to Test Drug Combination Against Neuroblastoma

Levy’s and Zweidler-McKay’s studies will require further research before testing the drugs in pediatric patients. However, Peter Zage, M.D., Ph.D., assistant professor at the Children’s Cancer Hospital, presented research on a drug that is currently available for young patients with neuroblastoma. Zage, who specializes in neuroblastoma, received this year’s Young Investigator Award from ASPHO.

By combining a novel multi-kinase inhibitor, vandetanib, with 13-cis-retinoic acid (CRA), a drug often used for severe acne, Zage found that the two therapies reduced neuroblastoma tumors by 86 percent in pre-clinical tests. Currently, there is a Phase I clinical trial open for children with multiple-relapsed neuroblastoma to further study the new therapy combination. This trial is the first in the world to test vandetanib in children.

Mauricio Ghisoli, M.D., presented research at ASPHO related to the most common childhood cancer, leukemia. Ghisoli, a fellow at the Children’s Cancer Hospital, discovered a cancer agent that can turn off a certain receptor that promotes the growth of leukemia cells.

In pre-clinical tests, the drug, AZ23, led to a 50 percent reduction of acute myelogenous leukemia cells in mice with no immune system. When studied in mice with an immune system present, researchers found that 60 percent of the mice treated with AZ23 had long-term survival with no presence of leukemia cells.

Selected Presentations Show Depth Of Children's Cancer Hospital Research Program

Additional research from the Children’s Cancer Hospital was also presented at the ASPHO meeting. The increase in studies that were selected for ASPHO mirrors the emphasis the hospital puts on developing new therapies for children that will be more efficient against cancer while being less toxic to pediatric patients.